Preclinical modeling highlights the therapeutic potential of hematopoietic stem cell gene editing for correction of SCID-X1

CRISPR-Cas9 gene repair of hematopoietic stem cells from patients with X-linked chronic granulomatous disease

(2017) Suk See De Ravin et al.   Science Translational Medicine

Limiting Thymic Precursor Supply Increases the Risk of Lymphoid Malignancy in Murine X-Linked Severe Combined Immunodeficiency

(2017) Samantha L. Ginn et al.   Molecular Therapy-Nucleic Acids

Targeted gene editing restores regulated CD40L function in X-linked hyper-IgM syndrome

(2016) Nicholas Hubbard et al.   BLOOD

Long-term multilineage engraftment of autologous genome-edited hematopoietic stem cells in nonhuman primates

(2016) C. W. Peterson et al.   BLOOD

CRISPR/Cas9 β-globin gene targeting in human haematopoietic stem cells

(2016) Daniel P. Dever et al.   NATURE

Non-genotoxic conditioning for hematopoietic stem cell transplantation using a hematopoietic-cell-specific internalizing immunotoxin

(2016) Rahul Palchaudhuri et al.   NATURE BIOTECHNOLOGY

Targeted gene addition in human CD34+ hematopoietic cells for correction of X-linked chronic granulomatous disease

(2016) Suk See De Ravin et al.   NATURE BIOTECHNOLOGY

Lentiviral hematopoietic stem cell gene therapy for X-linked severe combined immunodeficiency

(2016) Suk See De Ravin et al.   Science Translational Medicine

Multi-reporter selection for the design of active and more specific zinc-finger nucleases for genome editing

(2016) Benjamin L. Oakes et al.   Nature Communications

Highly Efficient Genome Editing of Murine and Human Hematopoietic Progenitor Cells by CRISPR/Cas9

(2016) Michael C. Gundry et al.   Cell Reports

Gene therapy returns to centre stage

(2015) Luigi Naldini   NATURE

Homology-driven genome editing in hematopoietic stem and progenitor cells using ZFN mRNA and AAV6 donors

(2015) Jianbin Wang et al.   NATURE BIOTECHNOLOGY

Stem Cell-Specific Mechanisms Ensure Genomic Fidelity within HSCs and upon Aging of HSCs

(2015) Bettina M. Moehrle et al.   Cell Reports

Chemokine treatment rescues profound T-lineage progenitor homing defect after bone marrow transplant conditioning in mice

(2014) S. L. Zhang et al.   BLOOD

CRISPR-Cas9 Knockin Mice for Genome Editing and Cancer Modeling

(2014) Randall J. Platt et al.   CELL

Lin-Sca1+Kit- Bone Marrow Cells Contain Early Lymphoid-Committed Precursors That Are Distinct from Common Lymphoid Progenitors

(2014) R. Kumar et al.   JOURNAL OF IMMUNOLOGY

Cell competition is a tumour suppressor mechanism in the thymus

(2014) Vera C. Martins et al.   NATURE

Targeted genome editing in human repopulating haematopoietic stem cells

(2014) Pietro Genovese et al.   NATURE

Promoterless gene targeting without nucleases ameliorates haemophilia B in mice

(2014) A. Barzel et al.   NATURE

GUIDE-seq enables genome-wide profiling of off-target cleavage by CRISPR-Cas nucleases

(2014) Shengdar Q Tsai et al.   NATURE BIOTECHNOLOGY

A guide to genome engineering with programmable nucleases

(2014) Hyongbum Kim et al.   NATURE REVIEWS GENETICS

Transplantation Outcomes for Severe Combined Immunodeficiency, 2000–2009

(2014) Sung-Yun Pai et al.   NEW ENGLAND JOURNAL OF MEDICINE

A Modified γ-Retrovirus Vector for X-Linked Severe Combined Immunodeficiency

(2014) Salima Hacein-Bey-Abina et al.   NEW ENGLAND JOURNAL OF MEDICINE

The new frontier of genome engineering with CRISPR-Cas9

(2014) J. A. Doudna et al.   SCIENCE

DNA targeting specificity of RNA-guided Cas9 nucleases

(2013) Patrick D Hsu et al.   NATURE BIOTECHNOLOGY

A Differentiation Checkpoint Limits Hematopoietic Stem Cell Self-Renewal in Response to DNA Damage

(2012) Jianwei Wang et al.   CELL

Editing T cell specificity towards leukemia by zinc finger nucleases and lentiviral gene transfer

(2012) Elena Provasi et al.   NATURE MEDICINE

Brain conditioning is instrumental for successful microglia reconstitution following hematopoietic stem cell transplantation

(2012) A. Capotondo et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA

Lentiviral-mediated gene therapy leads to improvement of B-cell functionality in a murine model of Wiskott-Aldrich syndrome

(2011) Marita Bosticardo et al.   JOURNAL OF ALLERGY AND CLINICAL IMMUNOLOGY

In vivo genome editing restores haemostasis in a mouse model of haemophilia

(2011) Hojun Li et al.   NATURE

Site-specific integration and tailoring of cassette design for sustainable gene transfer

(2011) Angelo Lombardo et al.   NATURE METHODS

Generating the optimal mRNA for therapy: HPLC purification eliminates immune activation and improves translation of nucleoside-modified, protein-encoding mRNA

(2011) Katalin Karikó et al.   NUCLEIC ACIDS RESEARCH

A self-inactivating lentiviral vector for SCID-X1 gene therapy that does not activate LMO2 expression in human T cells

(2010) S. Zhou et al.   BLOOD

Hematopoietic Stem Cell Quiescence Promotes Error-Prone DNA Repair and Mutagenesis

(2010) Mary Mohrin et al.   Cell Stem Cell

Insertional oncogenesis in 4 patients after retrovirus-mediated gene therapy of SCID-X1

(2008) Salima Hacein-Bey-Abina et al.   JOURNAL OF CLINICAL INVESTIGATION

Platelets prevent IFN- / -induced lethal hemorrhage promoting CTL-dependent clearance of lymphocytic choriomeningitis virus

(2008) M. Iannacone et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA